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beta thalassaemia

This page shows the latest beta thalassaemia news and features for those working in and with pharma, biotech and healthcare.

GSK hands rare disease assets over to Orchard

GSK hands rare disease assets over to Orchard

Along with Strimvelis, GSK is passing over two therapies in late-stage clinical development for metachromatic leukodystrophy (MLD) and Wiskott Aldrich syndrome (WAS), and one clinical programme for beta thalassaemia.

Latest news

  • Novo Nordisk pays $400m for EpiDestiny’s sickle cell drug Novo Nordisk pays $400m for EpiDestiny’s sickle cell drug

    Novo Nordisk has bolstered its biopharma unit with a global licensing deal for a small-molecule drug for genetic blood disorders sickle cell disease and beta thalassaemia. ... And as an oral, once-daily drug EpiDestiny’s candidate could be easier to

  • Third time lucky? Sanofi cuts an $11.6bn deal to buy Bioverativ Third time lucky? Sanofi cuts an $11.6bn deal to buy Bioverativ

    Buying Bioverativ does give it a stake in advanced approaches for haematological diseases however, as the biotech has an established collaboration with Sangamo Therapeutics looking at genome-editing approaches to beta ... thalassaemia and sickle cell

  • Mixed news for gene therapies at ASH conference Mixed news for gene therapies at ASH conference

    severe beta-thalassaemia (BT), a genetic disorder that causes severe anaemia and organ damage.

  • Biogen Idec taps Sangamo for blood disorder drugs Biogen Idec taps Sangamo for blood disorder drugs

    cell disease (SCD) and beta thalassaemia, and up to $300m in development and commercialisation milestones. ... blood. There are estimated to be around 90, 000 people with SCD in the US and another 2, 000 with beta thalassaemia.

  • Bursting the banks

    To determine whether deferiprone and deferoxamine could be used successfully as a combination therapy, a study was carried out in Greece in 36 patients with beta-thalassaemia. ... III trial in 586 patients with beta-thalassaemia and transfusion-related

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